Hear the voice of those with rare disorders

Rare
disease patients and their support groups are calling on the
government to put in place an orphan drugs access programme,
similar to those in place or being developed in most other
OECD countries.

They say that Pharmac is unable to
manage access to specialised medicines under its current
framework, and are calling for separate management of these
medicines in the Ministry of Health, just like the separate
process in place now for very specialised and high cost
medical and surgical treatments.

The omission of
fairness and equity from Pharmac’s policy and decisions
relating to specialised medicines is regarded by the groups
as a serious conflict with the rights of the patients and at
variance with health legislation, health strategies, and
Ministry of Health guidelines for decision making, which all
place equity, fairness and community values into decision
criteria.

NZORD and the support groups endorse
Pharmac’s core role of getting a good deal on medicines
for the bulk of the population, but say that Pharmac’s
track record regarding medicines for orphan diseases is an
appalling failure. “We think they should be left to get on
doing what they can do well, and have their failure remedied
by removing it from their responsibility,” the groups
say.

Thursday 28 February is Rare Disease Day and
draws attention to the needs of those with rare disorders.
NZORD, the NZ Organisation for Rare Disorders, is hosting a
seminar today at which several patients will be talking
about their difficulties getting access to new treatments
for rare “orphan” diseases. The groups’ concerns are
reinforced by presentations from health researcher Greg
Coyle who will discuss the findings of his PhD thesis which
examined Pharmac’s exceptional circumstances scheme, and
lawyer Lucy Elwood will be discussing legal aspects of the
issues.

John Forman, executive director of NZORD
says “These patients are being abandoned by the health
system. There is a systems failure in this area, as the
moral factors of fairness, equity and community values are
not being included in Pharmac’s decisions on specialised
medicines.”

“The 2011 review of the exceptional
circumstances scheme and the introduction of the new NPPA
scheme, actually made it more difficult for rare disease
patients to get a fair hearing,” says Forman. “The
criteria changed so rarity was no longer a prerequisite, but
they also made it practically an exclusion criteria”, he
said.

The criteria reflect Pharmac’s narrow
health economics and budget management approach, and exclude
the very important moral factors of equity, fairness, and
community values. It appears to be a deliberate exclusion of
moral considerations from decision making.

In
trying to find solutions and answers to the way in which
access to “orphan” medicines is managed, NZORD and a
number of associated support groups have been sent around in
circles. After a test case under the new system, which was
rejected by Pharmac as not eligible for consideration, they
approached Ministers for an examination of the policy
framework, but were ‘fobbed off’. They then approached
the Health Select Committee who directed them to the Health
Minister. The Minister initiated a report from the Ministry
of Health into the groups’ concerns, but before briefing
with the Ministry were even completed the Minister wrote to
say he could not interfere with funding decisions by
Pharmac, despite the request being about policy
accountability, not a specific funding decision. The
Minister then referred the matter to Pharmac which resulted
in a bland avoidance letter from Pharmac.

Unable to
get any traction from these approaches, the groups submitted
a case to the Ombudsman in October 2012, claiming unfair
application of the policy to an individual case, and
suggesting that the policy itself was not fairly formed. The
case is still being investigated by the Ombudsman and a
response is expected soon.

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